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Congenital, familial and genetic disorders – page 46

  • 796 clinical trials
  • 308 recruiting
  • 374 conditions
  • 27 countries

Conditions in this area

374 conditions with trials, the busiest first

Duchenne muscular dystrophy28Factor VIII deficiency25Sickle cell disease25Cystic fibrosis24Hereditary angioedema22Spinal muscular atrophy17Hypertrophic cardiomyopathy16Type IIa hyperlipidaemia15Glycogen storage disease type II14Factor IX deficiency13Sickle cell anaemia13Congenital cystic kidney disease12Huntington's disease12Myotonic dystrophy12Non-small cell lung cancer12Osteochondrodysplasia12Adrenogenital syndrome11Alpha-1 antitrypsin deficiency11K-ras gene mutation11Retinitis pigmentosa11Severe myoclonic epilepsy of infancy11Thalassaemia11Fabry's disease10Hereditary angioedema with C1 esterase inhibitor deficiency10+350

Clinical trials in Congenital, familial and genetic disorders

796 trials across Europe, recruiting first

Type of trial
Recruitment status
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796 clinical trials

Metachromatic leukodystrophy

Not recruiting

Study on Gene Therapy with Atidarsagene Autotemcel for Patients with Metachromatic Leukodystrophy

Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years
  • Substances:Atidarsagene Autotemcel
  • Sponsor:Orchard Therapeutics (Europe) Limited

Spinal muscular atrophy

Not recruiting

Study of NMD670 tablets to improve muscle strength in adults with Type 3 spinal muscular atrophy who can walk

Investigational
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:Nmd670
  • Sponsor:NMD Pharma A/S

Neonatal alloimmune thrombocytopenia

Not recruiting

Study on the Safety and Effects of RLYB212 in Pregnant Women at Risk for Fetal and Neonatal Alloimmune Thrombocytopenia

InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:18–64 years
  • Substances:Anti-(Integrin Beta-3) Human Monoclonal Antibody
  • Sponsor:Rallybio Ipa LLC

Activated PI3 kinase delta syndrome

Not recruiting

Study of Leniolisib for Children Aged 4 to 11 with Activated PI3K Delta Syndrome

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Leniolisib Phosphate
  • Sponsor:Pharming Technologies B.V.

Choroidal dystrophy+1

Not recruiting

Long-term Safety and Efficacy Study of Gene Therapy for Choroideremia and X-Linked Retinitis Pigmentosa Using AAV2-REP1 and AAV8-RPGR in Previously Treated Patients

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:18–64 years
  • Substances:Timrepigene Emparvovec
  • Sponsor:Biogen Idec Research Limited

Metachromatic leukodystrophy

Not recruiting

Study on Long-Term Safety and Efficacy of Cebsulfase Alfa for Children with Late Metachromatic Leukodystrophy Receiving Intrathecal Enzyme Replacement

InvestigationalNo placebo
Countries:CzechiaCzechia
  • Participants:0–17 years
  • Substances:Cebsulfase Alfa
  • Sponsor:Shire Human Genetic Therapies Inc.

EGFR gene mutation+1

Not recruiting

Study on Amivantamab, Lazertinib, and Bevacizumab for Patients with Advanced EGFR-Mutant Non-Small Cell Lung Cancer After Treatment Progression

Registered drugNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Amivantamab
  • Sponsor:ETOP IBCSG Partners Foundation

Corneal dystrophy

Not recruiting

Study on the Effectiveness and Safety of STN1010904 Eye Drops for Patients with Fuchs Endothelial Corneal Dystrophy

Investigational
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Sirolimus
  • Sponsor:Santen Inc.

Duchenne muscular dystrophy

Not recruiting

Study on Long-Term Safety and Effectiveness of Vamorolone in Boys with Duchenne Muscular Dystrophy Who Completed Previous Vamorolone Studies

Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:Vamorolone
  • Sponsor:Santhera Pharmaceuticals (Schweiz) AG

Metastatic neoplasm+1

Not recruiting

Study on Treating Advanced Cancers with BRAF Mutations Using Sorafenib and Trametinib for Patients with Impaired Kinase Activity

Registered drugNo placebo
Countries:GermanyGermany
  • Participants:18–64 years · 65+ years
  • Substances:Sorafenib
  • Sponsor:Medical Center - University Of Freiburg

Sickle cell disease

Not recruiting

Study of Mitapivat for Patients with Sickle Cell Disease and Kidney Disease

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Mitapivat
  • Sponsor:Agios Pharmaceuticals Inc.

Rett syndrome

Not recruiting

Study on the Safety and Tolerability of Leriglitazone for Children with Rett Syndrome

Investigational
Countries:SpainSpain
  • Participants:0–17 years
  • Substances:Leriglitazone
  • Sponsor:Minoryx Therapeutics S.L.

Substances studied in this area

The therapies tested most often in Congenital, familial and genetic disorders trials.

Sirolimus21 clinical trialsSodium Chloride21 clinical trialsPrednisolone12 clinical trialsPlerixafor11 clinical trialsBusulfan10 clinical trialsIvacaftor9 clinical trialsMitapivat9 clinical trialsParacetamol8 clinical trialsTezacaftor8 clinical trials
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